A Treatment for Inherited Metabolic Disorders is Being Trialled in Patients

A new treatment under development has produced promising results in patients with blood cancers and is now being tested in inherited metabolic disorders, reports Financial Buzz.

Inherited metabolic disorders are a category of diseases that are genetic and involve the metabolism – the process of converting food into energy. There are several hundred types of inherited genetic disorders, but some are more common than others. Examples of inherited metabolic disorders include Gaucher disease, Hunter syndrome, and Hurler syndrome. Usually the disease is caused by a genetic mutation that results in a specific enzyme not working or not being made. This means that the enzyme cannot fulfil its role and the body’s metabolic processes are disrupted, often resulting in a toxic build-up of a substance, or a missing substance that cannot be produced. This creates the symptoms of disease. Different inherited metabolic disorders often require different treatments, but unfortunately the options tend to be limited. However, although the disease mechanisms vary between conditions, there are several common types of treatment. These include reducing the intake of substances that the body can’t process, clearing toxic build-up of substances, and replacing the missing enzyme or other molecule artificially.

A possible new treatment, developed by Magenta Therapeutics, is called MGTA-456 and is being tested for effectiveness at treating inherited metabolic disorders. It is made using stem cells taken from an umbilical cord, which are combined with a protein called an aryl hydrocarbon receptor. Patients are then given the treatment through a bone marrow transplant. MGTA-456 is hoped to be a good treatment option for patients because it can use a relatively small amount of blood from an umbilical cord to produce large doses of the treatment for the patient. In addition to higher doses, the treatment tends to allow patients to be treated with a better biological donor match. These two factors are hoped to reduce the risk of complications from the umbilical cord blood transplantation, which can be life-threatening. They are also thought to make engraftment more likely to occur, and occur to earlier. Engraftment is the term used when transplanted blood producing cells start to grown and create healthy blood cells in the patient.

The potential treatment will be evaluated for it’s safety and effectiveness at treating patients with inherited metabolic disorders in a phase ii study. Researchers are enrolling patients from the University of Minnesota, and hope to work with twelve patients with ALD, Krabbe disease, metachromatic leukodystrophy, or Hurler’s syndrome. The researchers’ are planning to follow patients up to check the success of engraftment, and also the safety and reactions of the patients. The study listing on the US government clinical trials website can be found here.

After previous successful studies in patients with blood cancers, researchers are hopeful about the applicability of this therapy to those with inherited genetic disorders. The company, Magenta, also aims to investigate whether the treatment could be extended to other serious diseases in the future.

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